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This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants With ISM | Patients who are currently being treated or plan to be treated for ISM |
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| Measure | Description | Time Frame |
|---|---|---|
| Baseline Demographics | Baseline (Month 1) | |
| Change From Baseline in Patient-Reported Outcomes Scores | Baseline up to Month 61 | |
| Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF) | Baseline up to Month 61 | |
| Change From Baseline in Serum Tryptase | Baseline up to Month 61 | |
| Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies | Baseline up to Month 61 |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with ISM who are currently being treated or plan to be treated with symptom-directed therapies and/or avapritinib for ISM.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Blueprint Medicines | Contact | 1-888-258-7768 | medinfo@blueprintmedicines.com | |
| Blueprint Medicines, EU Contact | Contact | +31 85 064 4001 | medinfoeurope@blueprintmedicines.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill) | Recruiting | Chestnut Hill | Massachusetts | 02467 | United States |
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| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| D008415 | Mastocytosis |
| ID | Term |
|---|---|
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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With the participant's informed consent and as approved by local IRBs/IECs, de-identified biological samples may be stored at a biosample repository and may be shared with the data repository. These samples could be used to research the causes of ISM, its complications and other conditions for which individuals with ISM are at increased risk, and to improve treatment. The repository will also be provided with a code-link that will allow linking the biological specimens with the phenotypic data from each participant, maintaining the blinding of the identity of the participant.
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |